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Welcome to Gene Therapy Net

Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials. For resources pertaining to regenerative cell therapy and clinical treatment, check out R3 Stem Cell.

Gene Therapy Success in Hereditary Angioedema Patients

Posted on: 2 February 2024, source: Technologynetworks
A single dose of a CRISPR/Cas9 therapy has been successful in treating hereditary angioedema. A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher. The patients from New Zealand, the Netherlands and the UK have hereditary angioedema, a genetic disorder characterised by severe, painful and unpredictable swelling attacks. These interfere with daily life and can affect airways and prove fatal.
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FDA Approves CRISPR Gene Therapy to Treat Blood Disorder

Posted on: 24 January 2024, source: healthnews.com
The U.S. Food and Drug Administration (FDA) has approved CRISPR gene editing cell therapy to treat a rare inherited blood disorder called transfusion-dependent beta-thalassemia (TDT). CRISPR Therapeutics and Vertex Pharmaceuticals’ gene editing cell therapy, branded as Casgevy, has officially been approved to treat TDT — a rare inherited blood disorder that requires regular blood transfusions — in patients 12 and over. Vertex announced Tuesday that it had been given FDA approval more than two months ahead of its original expected action date, and just over a month after the same treatment to treat sickle cell disease was approved.
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FDA Signals Imminent Approval for Sarepta’s Duchenne Gene Therapy: A Major Breakthrough for Patients

Posted on: 12 January 2024, source: medriva.com
The world of gene therapy is on the cusp of a significant milestone. Peter Marks, the Food and Drug Administration’s (FDA) top regulator of gene therapies, has indicated the full approval of Sarepta’s Duchenne gene therapy, Elevidys, is imminent. This development has the potential to positively impact patients, caregivers, and the healthcare industry as a whole.
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Revolutionary gene therapy shows promise in treating autism

Posted on: 6 December 2023, source: Earth.com
Chinese scientists have achieved a major advancement in gene therapy for autism, as reported in the journal Nature Neuroscience. This innovative therapy, developed by a research team in Shanghai, has been successfully tested on mice. The research marks a significant step in the treatment of autism spectrum disorder (ASD) through genetic base editing within the brain.
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Cell & Gene Therapy Space Gears Up for First CRISPR Approval

Posted on: 13 November 2023, source: Biospace
Halloween brings to mind pumpkins and trick or treat, but for the 100,000 Americans suffering from sickle cell disease, this past October 31 had a different meaning when an FDA advisory panel deemed the first potentially curative SCD therapy safe enough for clinical use, bolstering hopes of an FDA approval in December. It would also be the first-ever approval for CRISPR, the genetic modification technique that won its discoverers the Nobel Prize in 2020 for their 2012 breakthrough.
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Gene therapy restores hearing in children with rare form of deafness

Posted on: 28 October 2023, source: Science
Several deaf children can hear after receiving gene therapy—a first for the approach—a team at Fudan University reported today at a meeting in Belgium. The children were born deaf because they inherited two defective copies of the gene for a protein called otoferlin that helps the inner ear’s hair cells transmit sound to the brain. In an attempt to restore this function, researchers injected harmless viruses carrying DNA for a working copy of the otoferlin gene into the children’s ears. Four of five patients treated now have some hearing, the MIT Technology Review reports.
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UK biotech AlveoGene launches with plans for inhaled gene therapy

Posted on: 16 September 2023, source: BiopharmaDive
The company aims to capitalize on research by the Respiratory Gene Therapy Consortium, and is working first on a treatment of alpha-1 antitrypsin deficiency. The unusual approach could be a way to more reliably deliver gene therapies directly to the epithelial cells of the lungs. The company said preclinical research suggests its technology — dubbed InGenuiTy — could work with high efficiency and long-lasting effects. AlveoGene aims to bring its first candidate, a treatment for patients with alpha-1 antitrypsin deficiency or AATD, into clinical testing over the next two to three years. The company said it will also look to combine its platform with other technologies to target additional diseases such as lung surfactant deficiencies and idiopathic pulmonary fibrosis.
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